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Articles

Safety in Lactation: Corticosteroids

9 November 2020There is very limited information on the use of corticosteroids during breastfeeding, although they are likely to be present in milk. Avoid prolonged high dose…
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Medicine Compliance Aid Stability

CalcortSanofi

Sanofi
Calcort
Tablets 6mg
R2 · Red 2Drug is not suitable for CAs due to theoretical reasons that cannot be mitigated.
Unsuitable
Unsuitable.
21 November 2012

Lactation Safety Information

Prednisolone
No published evidence of safety
Used in infants >1 month
Doses up to 60mg daily unlikely to cause adverse effects in the infant
6 August 2020

New Medicines

EmflazaDuchenne muscular dystrophy

Information

Emflaza
New formulation
Not Known
PTC Therapeutics

Development and Regulatory status

None
None
Launched
Yes
01. Aug 13: FDA grants Orphan Drug designation [4].
02. Jan 15: US FDA grants Fast Track designation for deflazacort in treatment of patients with DMD. FDA previously granted Orphan Drug designation [1].
03. Jan 15: US filing anticipated in early 2016 [1].
04. Oct 15: FDA grants Rare Paediatric Disease designation [4].
05. Apr 16: PIII development ongoing in US; New Drug Application planned for May 2016: no indication of activity in EU [5].
06. Jun 16: Filed in the US. Deflazacort will now be assessed by the FDA as to whether it will accept the filing. This process typically takes around 2 months. The NDA filing is supported by both preclinical and clinical study programs, including two pivotal efficacy trials licensed by Marathon in more than 200 Duchenne patients 5 to 15 years of age. There is also an expanded access program, called Access DMD, ongoing in the U.S. that gives deflazacort to patients with Duchenne free of charge during the NDA review process [7].
07. Aug 16: FDA grants priority review to NDA for deflazacort for DMD [8].
08. Feb 17: FDA approves Emflaza (deflazacort) tablets and oral suspension for treatment of DMD in patients aged ≥ 5 years [10].
09. May 17: In the US, PTC Therapeutics have purchased Emflaza (deflazacort) from Marathon and plan to launch it at a much lower price [12]
10. Aug 17: PTC Therapeutics have successfully launched deflazacort immediate-release tablets (6mg, 18mg, 30mg and 36mg) and immediate-release oral suspension (22.75 mg/mL) in US with establishment of EMFLAZACares Program [13].
11. May 18: PTC does not have any current plans to file for approval of Emflaza (deflazacort) in the UK [14].

Category

A glucocorticoid with anti-inflammatory and immunosuppressant properties. Immediate-release tablets (6mg, 18mg, 30mg and 36mg) and immediate-release oral suspension (22.75 mg/mL) formulation.
About 1 in 3,500 boys in the UK are born with DMD [3].
Duchenne muscular dystrophy
Oral

Trial or other data

01. Deflazacort tablets are available in several countries worldwide since the 1970s; however, the product is not approved in the US [1].
02. Nov 14: Marathon Pharmaceuticals initiates a PI study to determine how multiple doses of rifampin, a CYP3A4 inducer, and clarithromycin, a CYP3A4 inhibitor, would affect the pharmacokinetics of a single dose of deflazacort, in healthy volunteers (NCT02286635). The study will enrol approximately 58 subjects, aged 18-80 years, in the US [2].
03. Dec 14: Four trials start. An open-label US PI trial to assess the effect of renal impairment on the pharmacokinetics of deflazacort (MP-104-CL-024; NCT02286622). The trial will compare the pharmacokinetics of the active moiety, 21 desacetyl DFZ and the prodrug deflazacort, in 16 healthy volunteers and subjects with renal impairment. A PI study to evaluate the pharmacokinetics and safety of once-daily oral deflazacort in children and adolescents with Duchenne muscular dystrophy (MP-104-CL-005; NCT02251600). The study is expected to enrol approximately 24 subjects aged 4-16 years, in the US. A long-term extension trial in patients who participated in the MP-104-CL-005 trial (NCT02295748). A PI trial to determine the effect of moderate (Child Pugh grade B) hepatic impairment on the pharmacokinetics of deflazacort (NCT02286609; MP-104-CL-023). This open-label, single dose evaluation trial intends to enrol 16 subjects in the US [1].
04. Apr 16: Marathon Pharma provide further detail on the previously presented primary endpoint of the study (n=196), which showed deflazacort improved muscle strength in DMD patients versus placebo at 12 weeks (p=0.0173 and p=0.0003 for 0.9 mg/kg/d and 1.2 mg/kg/d doses, respectively) [6].
05. Sep 16: RCT (n=196) found prednisone (PD) and both doses (0.9 and 1.2 mg/kg/d) of deflazacort (DZ) improved muscle strength from baseline vs. placebo at 12 weeks, but DZ was linked to less weight gain vs. PD. At 52 weeks, PD group had significantly more weight gain than DFZ groups [9].